- Conditions
- Fabry Disease
- Interventions
- Interview
- Other
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Not listed
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 529–552
of 1,709
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2034
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Jacksonville, Florida • Atlanta, Georgia + 10 more
- Conditions
- Huntington's Disease
- Interventions
- Physical Activity Coaching
- Other
- Lead sponsor
- Teachers College, Columbia University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Anemia of Prematurity
- Interventions
- Red blood cell transfusion
- Procedure
- Lead sponsor
- McMaster University
- Other
- Eligibility
- Up to 48 Hours
- Enrollment
- 424 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2005
- U.S. locations
- 2
- States / cities
- Albany, New York • Brooklyn, New York
- Conditions
- Type 1 Gaucher Disease
- Interventions
- AT2101
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- Miramar, Florida
- Conditions
- Argininosuccinic Aciduria, Argininosuccinate Lyase Deficiency, Urea Cycle Disorders
- Interventions
- Nitric oxide supplement, Placebo
- Dietary Supplement
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 8 Years to 64 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
- Interventions
- AJ201, Placebo
- Drug
- Lead sponsor
- AnnJi Pharmaceutical Co., Ltd.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 6
- States / cities
- Orange, California • Palo Alto, California • Jacksonville, Florida + 3 more
- Conditions
- Tuberous Sclerosis Complex, Epilepsy
- Interventions
- Sirolimus, Placebo
- Drug
- Lead sponsor
- Darcy Krueger
- Other
- Eligibility
- 1 Day to 6 Months
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 8 more
- Conditions
- Lower Urinary Tract Symptoms
- Interventions
- Lactobacillus
- Drug
- Lead sponsor
- Medstar Health Research Institute
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Angelman Syndrome
- Interventions
- GTX-102, Sham-LP
- Drug · Procedure
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 4 Years to 17 Years
- Enrollment
- 129 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 11 more
- Conditions
- Huntington's Disease
- Interventions
- Minocycline
- Drug
- Lead sponsor
- FDA Office of Orphan Products Development
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 63 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2003
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD, Muscular Dystrophy
- Interventions
- VM100 Nutritional Supplement
- Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 6 Years and older · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Unverricht-Lundborg Disease
- Interventions
- Placebo, BRV 2.5 mg, BRV 25 mg, BRV 50 mg
- Other · Drug
- Lead sponsor
- UCB Pharma
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 4
- States / cities
- San Francisco, California • Gainesville, Florida • New York, New York + 1 more
- Conditions
- Gaucher Disease Type 1
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 16 Years to 90 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Brain and Central Nervous System Tumors, Kidney Cancer, Liver Cancer, Retinoblastoma, Sarcoma
- Interventions
- filgrastim, busulfan, etoposide, ifosfamide, melphalan, thiotepa, stem cell transplantation, tomotherapy, total marrow irradiation, Mesna, Whole lung radiation
- Biological · Drug · Procedure + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2016
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Phenylketonuria
- Interventions
- Kuvan, Meal Challenge
- Drug · Other
- Lead sponsor
- Emory University
- Other
- Eligibility
- 10 Years to 45 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Tourette Syndrome, Chronic Tic Disorder, Oppositional Defiant Disorder, ADHD
- Interventions
- Anger control training
- Behavioral
- Lead sponsor
- Yale University
- Other
- Eligibility
- 11 Years to 16 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2006
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- Conditions
- Pompe Disease
- Interventions
- BMN 701
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 4
- States / cities
- San Diego, California • Gainesville, Florida • Tampa, Florida + 1 more
- Conditions
- Tourette Syndrome, Tic Disorders
- Interventions
- Habit reversal therapy, Supportive therapy
- Behavioral
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 16 Years and older
- Enrollment
- 122 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2010
- U.S. locations
- 3
- States / cities
- New Haven, Connecticut • Boston, Massachusetts • San Antonio, Texas
- Conditions
- Pelizaeus-Merzbacher Disease
- Interventions
- ION356
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 17 Years · Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 3
- States / cities
- Atlanta, Georgia • Philadelphia, Pennsylvania • Salt Lake City, Utah
- Conditions
- Gaucher Disease, Type 1
- Interventions
- velaglucerase alfa
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 3 Years and older
- U.S. locations
- 32
- States / cities
- Phoenix, Arizona • Beverly Hills, California • La Jolla, California + 28 more
- Conditions
- Huntington Disease
- Interventions
- Nilotinib 150 MG
- Drug
- Lead sponsor
- Georgetown University
- Other
- Eligibility
- 25 Years to 90 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Wilson Disease
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Miami, Florida • Chicago, Illinois + 2 more
- Conditions
- Hypopituitarism, Hypogonadism, Thyroid Dysfunction, Bone Diseases, Metabolic
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 2 Years to 24 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2018
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland